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. 2025 Mar;114(3):508-516.
doi: 10.1111/ejh.14357. Epub 2024 Dec 5.

ORPHEE: A Real-World Study on rIX-FP Prophylaxis Use in Adolescent/Adult Patients With Hemophilia B

Fabienne Volot  1 , Sabine Castet  2 , Alexandra Fournel  3 , Birgit Frotscher  4 , Benjamin Gillet  5 , Dominique Desprez  6 , Brigitte Tardy  7   8 , Antoine Rauch  9 , Pierre Chamouni  10 , Christine Biron-Andreani  11 , Jean-Baptiste Valentin  12 , Annie Harroche  13 , Yesim Dargaud  14   15 , Brigitte Pan Petesch  16 , Roseline d'Oiron  17   18 , Claire Berger  7   19 , Claire Reynes  20 , Thomas Lauvray  21 , Emmanuelle de Raucourt  22   23 , Abel Hassoun  24 , Aurélien Lebreton  25 , Vincent Cussac  26 , Hasan Catovic  27 , Cédric Martin  27 , Benoit Guillet  28   29
Affiliations

ORPHEE: A Real-World Study on rIX-FP Prophylaxis Use in Adolescent/Adult Patients With Hemophilia B

Fabienne Volot et al. Eur J Haematol. 2025 Mar.

Abstract

Objectives: To assess the real-world efficacy and safety of recombinant factor IX albumin fusion protein (rIX-FP) in patients with hemophilia B (HB) in France.

Methods: Data on dosing frequency, weekly consumption, and bleeds before-and-after switching to rIX-FP, were collected from December 2021 to February 2024. Annualized (spontaneous) bleeding rates [A(s)BRs] were calculated only in patients on prophylaxis with a follow-up ≥ 6 months.

Results: This interim analysis focused on 77 patients ≥ 12 years; 62 (81%) had severe HB. After switching to rIX-FP, the infusion interval was 14 (7-14) days. Weekly consumption was 43 (35.5-53) IU/kg. ABRs and AsBRs were 0.5 (0-1.9) and 0 (0-0.7) (n = 63) at 18.2 (12.3-21.9) months of follow-up. Prophylactic efficacy of rIX-FP was considered 'Excellent'/'Good' in 65/68 (95%) patients. Among the 43 patients previously treated with rFIXFc, 21 increased the infusion interval from 7 (7-11) days with rFIXFc to 14 (7-14) days with rIX-FP; 33/43 (77%) reduced weekly factor IX (FIX) consumption from 59.95 (46.35-77.93) to 42.5 (35.88-50.25) IU/kg. Patients maintained good protection against bleeds.

Conclusion: This analysis confirmed that switching to rIX-FP allows for reducing injection frequency and FIX consumption while maintaining good bleed protection.

Keywords: efficacy; hemophilia B; prophylaxis; rFIXFc; rIX‐FP.

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Conflict of interest statement

Fabienne Volot: declares COI from Sobi, Roche, Pfizer, Takeda and CSL Behring. Sabine Castet: received fees as consultant or speaker from CSL Behring, LFB, Novo Nordisk, Sobi, and Roche‐Chugai. Alexandra Fournel: declares no COI. Birgit Frotscher: declares COI from Sobi, CSL Behring and Novo Nordisk. Benjamin Gillet: declares COI from Sobi, Roche, Roche‐Chugai, Sanofi, and Novo Nordisk. Dominique Desprez: declares COI from Sobi, Roche, Takeda and CSL Behring. Brigitte Tardy: declares COI from Sobi, Roche, CSL Behring, Novo Nordisk, Baxalta, Bayer and Pfizer. Antoine Rauch: received research support for his institution from CSL Behring and Roche‐Chugai, participated in clinical trials, advisory boards, and symposia for BioMarin, CSL Behring, LFB, Novo Nordisk, Octapharma, Roche‐Chugai, and Sobi. Pierre Chamouni: has been a consultant for Sobi. Christine Biron‐Andreani: received funding from CSL Behring, Takeda, Sobi, LFB, and Roche. Jean‐Baptiste Valentin: declares COI from CSL Behring, Octapharma, and Sobi. Annie Harroche: participated in clinical trials, advisory boards, and symposia for CSL Behring, Roche, Sobi, LFB, Bayer, Takeda, Octapharma, Novo Nordisk, and Sanofi. Yesim Dargaud: received grants/research support from Bayer, Baxter, Baxalta, Novo Nordisk, CSL Behring, LFB, Pfizer, LEO Pharma, Octapharma, and Stago; an educational grant from Takeda, and honoraria from Bayer, Baxter, Novo Nordisk, CSL Behring, Sobi and Octapharma. Brigitte Pan Petesch: has been a consultant for Sobi, CSL Behring, Takeda, BioMarin, Novo Nordisk and Roche‐Chugai. Roseline d'Oiron: has been a consultant for Bayer, Baxter/Baxalta/Shire/Takeda, BioMarin, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sobi and Spark Therapeutics. Claire Berger: declares COI for Sobi and Octapharma. Claire Reynes: declares COI from Novo Nordisk, CSL Behring and LFB. Thomas Lauvray declares no COI. Emmanuelle de Raucourt: declares COI from Sobi, LFB, CSL Behring, Roche and Takeda. Abel Hassoun: has been a consultant for Bayer, CSL Behring, and Sobi. Aurélien Lebreton: declares COI from Sobi, Takeda, LFB, Novo Nordisk, CSL Behring, Pfizer, Octapharma and Bayer. Vincent Cussac: declares COI from Takeda and Octapharma and is a Novo Nordisk employee. Hasan Catovic: declares CSL Behring employee. Cédric Martin: declares CSL Behring employee. Benoit Guillet: has been a consultant for Baxter/Baxalta/Shire/Takeda, CSL Behring, LFB, Novo Nordisk, Octapharma, Roche‐Chugai and Sobi.

Figures

FIGURE 1
FIGURE 1
Changes in annualized infusion rate and annualized FIX consumption in patients switching from rFIXFc (n = 43) to rIX‐FP for prophylaxis. Data are the mean ± standard error of the mean. ***p < 0.001 (Wilcoxon signed‐rank test). FIX, factor IX.
FIGURE 2
FIGURE 2
Radar plots summarizing the annualized infusion rate, weekly consumption (IU/kg/week), and percentage of patients with spontaneous or joint bleeds among those who switched from rFIXFc (n = 43) to rIX‐FP for prophylaxis.
See this image and copyright information in PMC

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